Unproven stem cell treatment cause for alarm

High-quality scientific research is hard work. There are moments of serendipity, to be sure, but even the most inspired ideas demand scrupulous validation. Nowhere is that more true than in the health sciences and medicine where our well-being and lives are literally at stake.

As stem cell scientists seeking new ways to treat many terrible diseases, we must take great care to ensure the therapies we develop are based upon sound scientific research, and proven to lack undesirable or life-threatening complications.

Yet as we write, the Italian Parliament is considering legalization of an unproven, untested and largely unexplained stem cell treatment. Good science and lives are at risk, with potential implications and consequences for vulnerable patients and families in San Diego.

The treatment in question involves extracting stem cells from bone marrow, manipulating them in the lab and then re-injecting the cells back into patients with severe neurological diseases. These altered stem cells are supposed to differentiate into new, healthy replacement neurons.

The company promoting the treatment once sold anti-cellulite treatments. None of the established standards, expectations and laws of science and good governance appears to apply: The method has never been described in a peer-reviewed scientific journal. There have been no multiphase clinical trials. Absolutely no one can assert with legitimate confidence that the treatment is safe, let alone that it actually works.

It is on the verge of government approval entirely as the result of a massive publicity effort fueled by money, celebrity endorsements, unsubstantiated claims and the power of emotion. Promoters say it’s a last chance for a group of terminally ill patients, many of them children. They argue legalization would be an act of compassion.

As scientists and doctors who study devastating diseases like Alzheimer’s, we are sometimes asked by patients why, when things seem hopeless, they shouldn’t resort to untested drugs or unproven therapies. “I’m already so badly off,” they say, “how could it be worse?”

It can always be worse.

An unproven drug or therapy may not kill, but what if it caused incurable pain? What if it did nothing at all but render your family bankrupt? Questionable stem cell treatments often cost tens of thousands of dollars, not just in faraway countries but across the border and in San Diego where they may be offered under various guises.

In Italy, scientists and physicians who oppose the stem cell treatment have been publicly excoriated as uncaring. This is an outrage and a lie. These people have devoted their lives and careers to finding better treatments and cures. They are human, with families and friends who may also be affected with terrible diseases. But they also recognize their responsibility to speak the truth, however uncomfortable or unpopular. There is no compassion in selling dubious treatments to the weak, sick and desperate.

In the United States, we have rules and laws for the development and testing of new drugs and therapies, overseen and enforced by agencies like the U.S. Food and Drug Administration. For the most part, these rules and laws work, though they require regular review and refinement, and must be constantly protected from erosive factors like underfunding and attempts to soften or remove specific protections.